Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,767 recruiting studies in this area right now.
By phase
- Not applicable2,684
- Phase 2280
- Phase 1241
- Phase 3210
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease134
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)38
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
Leading sponsors
- Assistance Publique - Hôpitaux de Paris57
- Mayo Clinic39
- Boston Children's Hospital38
- National Institute of Allergy and Infectious Diseases (NIAID)38
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Children's Hospital Medical Center, Cincinnati31
- Hospices Civils de Lyon29
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California324
- Maryland160
- Alabama133
- Massachusetts108
- Arizona87
- New York85
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,767 total
- NCT06245356RecruitingPhase 2
A safety trial of trifluridine/tipiracil for DPD-deficient patients
This trial tests a chemotherapy drug (trifluridine/tipiracil) in people with metastatic colorectal or gastroesophageal cancer who have a DPD deficiency. DPD deficiency means your body may have trouble breaking down certain chemotherapy drugs, which can lead to serious side effects. This study aims to see if this drug is safe and effective for you.
AmiensAges 18 years+ - NCT06239272RecruitingPhase 1/Phase 2
Personalized treatment for NRSTS in children and young adults
This trial tests a personalized treatment plan for children and young adults with a type of soft tissue sarcoma (NRSTS). It uses a combination of surgery, targeted radiation, and two drugs (pazopanib and selinexor) to fight cancer while reducing side effects. The study is looking for people who have not been treated before for this sarcoma.
Atlanta, GeorgiaAges Up to 30 years - NCT06240715Recruiting
Umbilical cord milking for premature babies who need help at birth
This study tests whether gently squeezing the umbilical cord before clamping it (called cord milking) helps premature babies who are not vigorous at birth. It is being done in hospitals with fewer resources to find safe and effective ways to improve oxygen and blood flow right after delivery.
Belagavi, KarnatakaAges birth–10 minutes - NCT04294043RecruitingPhase 1
Gallium IV study for adults with cystic fibrosis and NTM lung infection
This Phase 1 study tests whether an IV medicine called gallium can treat hard-to-treat lung infections (NTM) in adults with cystic fibrosis. It is mainly for people who have not been treated yet for NTM or who have NTM that has not cleared despite antibiotics.
Birmingham, AlabamaAges 18 years+ - NCT04569149Recruiting
Registry for very small-bodied, low-head-growth syndromes
This is a registry (a secure list of patients) for people with rare conditions that cause very small size and a smaller-than-usual head from early life. It helps researchers learn more about these conditions and how they vary from person to person.
Wilmington, DelawareAges Any age - NCT05519111RecruitingPhase 2
Cannabinoid treatment to lower inflammation and sickle pain
This Phase 2 study tests a cannabinoid medicine (dronabinol) to reduce inflammation and pain related to sickle cell disease. It may help you have less pain that interferes with daily life if you meet the study requirements and can safely avoid cannabis during the study.
New York, New YorkAges 18 years+ - NCT06222203Recruiting
Watching for cancer in NF1-related nerve tumors
This study monitors people with neurofibromatosis type 1 (NF1) over time to watch for signs that their nerve tumors might turn into cancer. It helps researchers learn which tumors are more likely to change and may lead to better care.
Bethesda, MarylandAges 3–120 - NCT06211348Recruiting
Genomic sequencing for healthy-looking fetuses
This study looks at the benefits of doing extra genetic testing (genomic sequencing) on samples from routine prenatal tests like CVS or amniocentesis, specifically for pregnancies where the baby appears normal on ultrasound.
San Francisco, CaliforniaAges 18–64 - NCT06219863Recruiting
Harness walking help for babies with Down syndrome
This study tests if a special harness can help babies with Down syndrome learn to walk earlier. It is designed for infants under 2 years old who can sit but are not yet walking.
Boston, MassachusettsAges Up to 2 years - NCT06213090Recruiting
Study on patterns of neurodevelopmental disorders
This study looks at patterns of brain development issues in children who visit a specific medical center. It may help doctors understand these conditions better.
Phoenix, ArizonaAges Any age - NCT06226051Recruiting
Growing Little PEAPODS Preterm Baby Study
This study follows the growth and body composition of very premature babies (born between 22 and 32 weeks) to learn how they develop. It uses a special device called the PEAPOD to measure body fat, which may help improve care for preemies.
Madison, WisconsinAges 5 months–7 months - NCT06222632Enrolling by invitation
MIND diet and forest bathing for heart and mind
This study tests whether a brain-healthy diet (MIND diet) combined with relaxing walks in nature (forest bathing) can help protect your heart and improve your mood. It is for Chinese adults aged 50–75 who have high cholesterol and some anxiety.
Hong KongAges 50–75 - NCT06218433Recruiting
Bladder cancer screening for Lynch syndrome with a urine test
This study is testing a urine test to see if it can find bladder cancer early in people with Lynch syndrome. If you have Lynch syndrome and are between 50 and 75, this screening could help catch cancer before it causes symptoms.
VancouverAges 50–75 - NCT06219421Recruiting
Using facial photos to understand genetic conditions
This study uses regular front and side facial photos to help doctors better understand facial features in people with genetic conditions or after surgery. It may help improve how we diagnose and care for these conditions.
ParisAges Any age - NCT06211504RecruitingPhase 4
Foot implant plus standard surgery for flatfoot
This study tests whether placing a small implant in the sinus tarsi (a space in the side of the foot) during a standard flatfoot surgery helps improve the results. It's for adults with stage II adult acquired flatfoot deformity who have not improved with conservative care.
Hässleholm, Skåne CountyAges 16–75 - NCT06216704Recruiting
Bone health and marrow study in teens with cystic fibrosis
This study looks at how cystic fibrosis affects bone health and bone marrow in teenagers. It uses a special MRI scan to measure bone strength and marrow composition, which may help doctors better understand and prevent bone problems in people with CF.
Boston, MassachusettsAges 13–20 - NCT06227780Recruiting
Sound therapy for thinking and sensory issues in kids with developmental disorders
This trial tests whether special sound patterns played through headphones can help improve thinking and reduce sensitivity to noise in children with Fragile X syndrome or autism. The study uses a short daily listening program over several weeks to see if it makes a difference.
Cincinnati, OhioAges 5–10 - NCT06210698Recruiting
Angioedema biomarker research study
This study collects blood samples (and optional skin samples) from people with angioedema to learn more about the condition and find better ways to diagnose and treat it. If you join, you may help scientists understand angioedema better.
Wheaton, MarylandAges 12–75 - NCT06217588Recruiting
LCAT Deficiency Natural History Study
This study tracks people with LCAT deficiency over time to learn more about how the condition affects the body. It does not test a treatment, but helps researchers understand the disease better so they can design future studies.
Philadelphia, PennsylvaniaAges Any age - NCT06222697Recruiting
Safety study of Jivi for hemophilia A in Korea
This study is checking the safety of Jivi (a Factor VIII medicine) when used in normal medical care for people with hemophilia A in Korea. It is for anyone 12 or older who has used Factor VIII before or is starting Jivi as part of their regular treatment.
Multiple LocationsAges 12 years+ - NCT06217861RecruitingPhase 1
Gene therapy for children with GA-1 not helped by standard care
This trial tests a new gene therapy (VGM-R02b) for children with Glutaric Acidemia Type 1 (GA-1) whose symptoms are not well controlled by standard treatment. The goal is to see if the therapy is safe and can help improve neurological symptoms.
Hangzhou, ZhejiangAges Up to 6 years - NCT05211570RecruitingPhase 1/Phase 2
Study drug AB8939 for AML after prior treatments
This Phase 1/2 study tests AB8939 in people with acute myeloid leukemia (AML) whose disease has come back or did not respond to earlier treatment. It may help researchers find a safe dose and learn whether the treatment can control the leukemia.
Houston, TexasAges 18 years+ - NCT06100614Recruiting
Using a blood test to reduce antibiotics in preterm newborns
This study checks if a blood test (presepsin) can help doctors safely stop antibiotics earlier in preterm babies at moderate risk of infection. The goal is to reduce unnecessary antibiotic use.
Amsterdam, North HollandAges Any age - NCT04342026Recruiting
Study environment and hormones in undescended testicles
This study looks at whether environmental exposures and hormone disruption are linked to undescended testicles in children. It may help researchers better understand causes and risk factors, which could improve future care for affected families.
MontpellierAges 1 month–18 years
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,767 recruiting medical genetics studies from the U.S. registry right now, out of 5,497 we cover in this area in total.
- What does “Genetic & congenital” cover here?
- Inherited and congenital conditions present from birth or written into the genes. We group each study into genetic & congenital based on the conditions it studies, so you can scan the whole therapeutic area at once and drill into a specific condition.
- How do I find a genetic & congenital trial near me?
- Use the search to filter genetic & congenital studies by location and eligibility. Every Clin2 trial page explains the criteria in plain English and offers a short, optional pre-screen for a fit read — the study team makes the final decision.
Other specialties
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov; specialty groupings are derived from each study’s conditions. Talk with your doctor about whether a specific trial is right for you.